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Raptor Pharmaceutical's PROCYSBI™ Receives FDA Approval For The Treatment Of Nephropathic Cystinosis

NOVATO, Calif., April 30, 2013 /PRNewswire/ -- Raptor Pharmaceutical Corp. ("Raptor" or the "Company") (Nasdaq: RPTP), today announced that the U.S. Food and Drug Administration (FDA) has approved PROCYSBI™ (cysteamine bitartrate) delayed release capsules for the treatment of nephropathic cystinosis in adults and children 6 years and older. In a Phase 3 study, PROCYSBI showed consistent cystine depletion over the full 12-hour dosing period. Sustained levels of cysteamine have not historically been achieved in the majority of patients in this population.  Studies have shown that sustained cystine depletion in patients may significantly delay disease progression, including kidney dysfunction, dialysis, kidney transplant, organ failure and premature death.

"PROCYSBI's approval advances Raptor closer to its goal of becoming an integrated, commercial-stage biopharmaceutical company. It is especially gratifying for us that patients and caregivers in the cystinosis community now have a long-anticipated treatment available to them," said Christopher M. Starr, Ph.D., CEO and co-founder of Raptor. "PROCYSBI is the result of a decade-long patient- and physician-initiated effort to improve the treatment and lives of cystinosis patients. We are very grateful to have worked together on the development, manufacturing and regulatory submission of this important treatment for the cystinosis community."

PROCYSBI is the culmination of early research funded by the patient advocacy group Cystinosis Research Foundation. The FDA approval of PROCYSBI was based on a New Drug Application (NDA) comprising data from six clinical trials, including a multi-center randomized, active-controlled Phase 3 trial of 43 patients with nephropathic cystinosis and extension data from that trial. The most commonly reported adverse reactions were vomiting, abdominal pain, discomfort, headaches, nausea, diarrhea, anorexia/decreased appetite, breath odor, fatigue, dizziness, skin odor and rash. Clinical studies are ongoing in children less than six years of age to evaluate for the first time the safety and efficacy of PROCYSBI. Over ninety percent of the cystinosis population is six years or older.

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